Spark Therapeutics Enters Collaboration With Pfizer To Develop New Hemophilia B Therapies

Spark Therapeutics has announced that it has entered into a global collaboration with Pfizer to develop and commercialize a potential treatment for hemophilia B, SPK-FIX, which is based on bio-engineered adeno-associated virus (AAV) vectors.

The goal of the collaboration is to bring an investigational therapy as soon as possible to patients with this rare but fatal inherited disease. The program is expected to enter early-stage clinical trials for hemophilia B in the first half of 2015.

Dr. Katherine High, President and CSO of Spark, who also pioneered the development of AAV-mediated gene therapy for hemophilia, said, "Pfizer's longtime experience in hemophilia, including strong relationships with physicians, patients, and payors, as well as clinical, regulatory, and commercial capabilities, will complement our team's deep knowledge of AAV-mediated gene transfer for the disease. We look forward to working with Pfizer with the goal of making gene therapy for hemophilia B a reality for patients."

According to the deal, Spark will get $20 million upfront from Pfizer and will also be eligible for additional milestone payments of about $260 million for development and commercialization goals. The deal covers multiple hemophilia B product candidates that may be developed under the collaboration.

For its part, Spark will be responsible for taking the therapy through the Phase 1/2 trials, while Pfizer will be responsible for pivotal studies, regulatory approvals, and potential global commercialization of the product. If the product is approved, Spark will also receive double-digit royalties based on global product sales.

Hemophilia B is a rare genetic blood disorder that causes severe bleeding and affects males more commonly. According to Spark, approximately 4,000 males in the U.S. and 26,000 males worldwide suffer from this condition. The currently available treatments are regular intravenous infusions of either plasma-derived or recombinant Factor IX, which helps with blood clotting.

Spark's bioengineered vectors are designed to deliver a high-activity Factor IX gene to patients, which would be effective for a number of years. Last month, Spark won the U.S. FDA’s Breakthrough Therapy Designation for its novel therapy, SPK-RPE65, for treating inherited night blindness. SPK-RPE65 uses the bioengineered vectors to target a group of blinding conditions known as inherited retinal dystrophies (IRDs).

Having developing the first recombinant Factor IX product for hemophilia, Pfizer also is a leader in this field, and would make an ideal partner for Spark’s hemophilia B program.

Pfizer senior VP and CSO of Rare Disease Research Unit, Dr. Kevin Lee said, "This agreement reinforces Pfizer's longstanding commitment to the hemophilia community. Spark's hemophilia B program has the potential to build on our leading hemophilia portfolio and could offer patients with this bleeding disorder a potential new treatment option."